Inherited neurological disorders affect both the central and peripheral nervous systems throughout the body, resulting in detrimental impacts on the patient's health and significant socioeconomic burdens on the healthcare system. Often there is no effective means to cure neurodegenerative diseases. Recent development in cell therapy and gene therapy have induced a paradigm shift in neurosciences, providing innovative solutions to develop treatments for inherited neurodegenerative diseases. Advances in pluripotent stem cells and cellular reprogramming technology provided a feasible approach to generate patient-specific cells in the lab as an unlimited cellular source for tissue engineering and regenerative medicine. In addition, numerous disease models have been developed using patient-specific stem cells to better understand disease pathogenesis, which also provided a personalized in vitro platform for drug discovery and test gene therapy. In the latter case, recent studies and on-going clinical trials demonstrated the promising potential of using gene therapy to correct the underlying genetic defects that caused the diseases. Ongoing research into the safety, specificity, and efficacy of gene therapy would be key to facilitate its translation from the bench to the clinics.
This article collection aims to provide an overview of the current advances in the development of cell therapy, regenerative medicine and gene therapy in treating neurological diseases, such as those affecting the brain, spinal cord or the eye. We welcome original studies, brief research report, methods/protocol papers, perspectives, reviews and mini-reviews related to the biological as well as the translational aspect of this topic. Topics to be covered include, but are not limited to the following:
- Development of gene delivery systems for clinical application of gene therapy
- Applications of CRISPR technology for gene therapy
- Cell therapy to treat neurological disorders: cell replacement and/or supportive trophic factors
- Cellular reprogramming and stem cell differentiation for in vitro generation of neural cells
- in vivo application of cellular reprogramming to stimulate neural regeneration
- Use of pluripotent stem cell models to study and develop the treatment for neural diseases
- Biomaterials to promote stem cell growth and differentiation
Inherited neurological disorders affect both the central and peripheral nervous systems throughout the body, resulting in detrimental impacts on the patient's health and significant socioeconomic burdens on the healthcare system. Often there is no effective means to cure neurodegenerative diseases. Recent development in cell therapy and gene therapy have induced a paradigm shift in neurosciences, providing innovative solutions to develop treatments for inherited neurodegenerative diseases. Advances in pluripotent stem cells and cellular reprogramming technology provided a feasible approach to generate patient-specific cells in the lab as an unlimited cellular source for tissue engineering and regenerative medicine. In addition, numerous disease models have been developed using patient-specific stem cells to better understand disease pathogenesis, which also provided a personalized in vitro platform for drug discovery and test gene therapy. In the latter case, recent studies and on-going clinical trials demonstrated the promising potential of using gene therapy to correct the underlying genetic defects that caused the diseases. Ongoing research into the safety, specificity, and efficacy of gene therapy would be key to facilitate its translation from the bench to the clinics.
This article collection aims to provide an overview of the current advances in the development of cell therapy, regenerative medicine and gene therapy in treating neurological diseases, such as those affecting the brain, spinal cord or the eye. We welcome original studies, brief research report, methods/protocol papers, perspectives, reviews and mini-reviews related to the biological as well as the translational aspect of this topic. Topics to be covered include, but are not limited to the following:
- Development of gene delivery systems for clinical application of gene therapy
- Applications of CRISPR technology for gene therapy
- Cell therapy to treat neurological disorders: cell replacement and/or supportive trophic factors
- Cellular reprogramming and stem cell differentiation for in vitro generation of neural cells
- in vivo application of cellular reprogramming to stimulate neural regeneration
- Use of pluripotent stem cell models to study and develop the treatment for neural diseases
- Biomaterials to promote stem cell growth and differentiation